Submitted by Anonymous (not verified) on 1 February 2024 - 11:06
Orphan designation: Humanised IgG1 monoclonal antibody against TfR1 conjugated to exon 44 specific phosphorodiamidate morpholino oligonucleotide via a non-cleavable linker Treatment of Duchenne muscular dystrophy, 13/10/2023 Positive
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